Research

Red blood cell deficiency or anemia is a debilitating condition often caused by failure to produce red blood cells. Today the only drugs used to increase red blood cell production in anemic patients are recombinant erythropoietin (Epo) analogues. Many types of anemia however do not respond to Epo treatment. Such Epo-resistant anemias, including aplastic anemias such as Diamond-Blackfan anemia (DBA), are today treated with blood transfusions. In this study I have set up three aims with the common goal to develop fundamentally new concepts for treating anemic patients that today lack effective drugs.

In Aim A I will identify new drug targets for promoting Epo-responsive progenitor production. The discoveries of novel pathways regulating red blood cell production will constitute fundamentally novel strategies for treatment of Epo-resistant anemia.

In Aim B I will identify drugs increasing red blood cell production in the EPO-resistant anemia DBA. This is an important step towards clinical drugs for DBA and possibly other anemias.

In Aim C I will reprogram skin cells to red blood cells. The invention of a method to generate red blood cells from skin constitutes a truly original approach to treat and study anemia and changes the way red blood cell development and anemia will be studied in the future.

To summarize this creative and challenging project will break new ground in development of novel therapeutic approaches for anemia.